Toward new drugs that turn genes on and off

June 4, 2009
Scientists found a group of molecules that could provide clues to the development of various diseases. Credit: The American Chemical Society

Scientists in Michigan and California are reporting an advance toward development of a new generation of drugs that treat disease by orchestrating how genes in the body produce proteins involved in arthritis, cancer and a range of other disorders. Acting like an “on-off switch,” the medications might ratchet up the production of proteins in genes working at abnormally low levels or shut off genes producing an abnormal protein linked to disease.

Their report is in the current issue of ACS .

In the study, Anna K. Mapp and colleagues discusses that cause to be active and churn out proteins — so-called transcriptional activators. That’s because because they control a key process known as transcription, in which instructions coded in genes produce proteins. Malfunctions in these activators could lead to altered transcription patterns that lead to disease. For example, variations in the tumor suppressor gene p53 are found in more than half of all human cancers.

Mapp describes discovery of a group of molecules that could be used to help scientists better understand transcription. Known as activator artificial transcriptional activation domains, these small molecules mimic natural activators and could provide insights on how mistakes in gene regulation result in various diseases.

“Evidence suggests that these small molecules mimic the function and mechanism of their natural counterparts and present a framework for the broader development of small molecule transcriptional switches,” Mapp states.

More information:

Source: ACS

Explore further: Newly identified mechanism for silencing genes points to possible anti-cancer strategies

Related Stories

Rewrite the textbooks: Transcription is bidirectional

January 25, 2009

Genes that contain instructions for making proteins make up less than 2% of the human genome. Yet, for unknown reasons, most of our genome is transcribed into RNA. The same is true for many other organisms that are easier ...

Small molecules mimic natural gene regulators

June 3, 2009

( -- In the quest for new approaches to treating and preventing disease, one appealing route involves turning genes on or off at will, directly intervening in ailments such as cancer and diabetes, which result ...

Recommended for you

A new form of real gold, almost as light as air

November 25, 2015

Researchers at ETH Zurich have created a new type of foam made of real gold. It is the lightest form ever produced of the precious metal: a thousand times lighter than its conventional form and yet it is nearly impossible ...

Getting under the skin of a medieval mystery

November 23, 2015

A simple PVC eraser has helped an international team of scientists led by bioarchaeologists at the University of York to resolve the mystery surrounding the tissue-thin parchment used by medieval scribes to produce the first ...


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.